CBER 2026 Orphan Approvals (new BLAs)
This list reflects information regarding the applications as of the approval date. It is not updated with regard to applicant or application status changes. The applications are listed by date of approval in reverse chronological order.
| Trade Name/Proper Name | Indication | Manufacturer/License Number | Approval Date |
|---|---|---|---|
| TREGZI | for use in matched donor hematopoietic stem cell transplantation (HSCT) with a myeloablative preparative regimen, for hematopoietic and immunologic reconstitution and to improve chronic graft-versus-host disease (cGHVD)-free survival, in the treatment of adults with hematological malignancies. | Orca Biosystems, Inc. | 6/30/2026 |
| OTARMENI | for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss (any frequency >90 dB HL) associated with molecularly confirmed biallelic variants in the OTOF gene, preserved outer hair cell function, and no prior cochlear implant in the same ear. | Regeneron | 4/23/2026 |
| KRESLADI | for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen (HLA)-matched sibling donor for allogeneic hematopoietic stem cell transplant. | Rocket Pharmaceuticals,RInc | 3/26/2026 |